Roche to license Ionis RNA-targeted Alzheimer's, Huntington's programs

Sept. 27, 2023

Ionis Pharmaceuticals has entered an agreement with Roche for two undisclosed early-stage programs for RNA-targeting investigational medicines for the treatment of Alzheimer's disease (AD) and Huntington's disease (HD). 

Under terms of the agreement, Ionis will advance the two programs through pre-clinical studies; Roche will take over the clinical development and, if approved, commercialization worldwide. The California-based biotech will receive a $60 million upfront payment from Roche and is eligible for milestone payments and tiered royalties.

The Ionis and Roche partnership dates back to a 2013 Huntington's disease alliance focused on tominersen, an Ionis-discovered investigational medicine for HD. The drug, designed to reduce the production of all forms of the huntingtin protein, is currently being evaluated by Roche in a phase 2 proof of concept study (GENERATION HD2) in people with prodromal or early manifest HD.

Ionis had a win earlier this year when the FDA approved Qalsody, developed in collaboration with Biogen, for the treatment of an ultra rare form of amyotrophic lateral sclerosis (ALS). The drug is the first approved treatment to target a genetic cause of ALS.